Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome

• Status: Recruiting
• Trial end date: 2022-07-01
• Target enrollment: 0
• Participant gender: All

Summary

A prospective non-randomized open label single arm clinical trial to examine the efficacy and safety of sirolimus in patients with Peutz-Jeghers Syndrome.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Peking Union Medical College Hospital
• Collaborators: Air Force General Hospital of the PLA; Chinese Academy of Medical Sciences; Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Patients are diagnosed with PJS.

• Patients have gastrointestinal polyps related syndromes, including abdominal pain, abdominal distension, gastrointestinal bleeding, etc, with imageological examination suggesting intestinal obstruction or intussusception; or whose symptoms recur after previous digestive endoscopic treatment and surgery; or who are inappropriate or unwilling to accept the above treatment again and wish to receive pharmacotherapy.

• Conventional treatment didn't work well in patients combined with PJS-related tumors.

• Physical condition (ECGO): 0~3

• Organ function is good and biochemical indices meet the following conditions:

• AST≤2.5×upper limit of normal value (ULN),

• ALT≤2.5×upper limit of normal value (ULN),

• Serum total bilirubin (TSB)≤1.5×upper limit of normal value (ULN),

• Creatinine≤1.5×upper limit of normal value (ULN).

• No other medications have been received for intestinal polyps within 3 months prior to the clinical trial.

• Patients participate in the trial voluntarily and have signed the informed consent by the participant or his/her legal guardian.

Exclusion Criteria:

• Patients underwent a surgery within 2 weeks.

• Patients may need emergency surgery in the near future.

• Patients are allergic to any ingredient of rapamycin.

• Patients suffer from a disease requiring immediate blood transfusion.

• Patients suffer from any disease or condition that may impact implementation of the study or interpretation of the results. This type of diseases includes:

• Known severe blood coagulation disorders

• Known anemia that is not caused by intestinal polyps

• Known hemoglobinopathy

• Other gastrointestinal infectious diseases

• Serious heart, liver, kidney and other concomitant diseases that may endanger lives

• Patients are in pregnancy and lactation.

• Alcohol or drug (such as aperient) abuse

• Patients took part in another clinical trial that may influence this study.

• The researchers believe that there are other unfavorable reasons for the patient to become a subject.